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Human Research Protection Program

Expanded access

Expanded access is a potential pathway to investigational drugs. This page will provide basic information on expanded access programs and the process to submit a request for emergency use* at Children's Wisconsin. For detailed information, including definitions, visit the FDA pages regarding expanded access – Information for physicians and the FDA expanded access - main page.

Expanded access, sometimes referred to as "compassionate use" is a potential, alternative pathway to get access to drugs or biologics that are otherwise unavailable for treatment, known as investigational products.

  • This is for treatment of a patient, not for an investigation within a clinical trial
  • This is for treatment of patients with an immediately life threatening condition or serious disease
  • The product is investigational, not available commercially
  • The product is not available through any clinical trials (e.g., patient ineligibility, lack of ongoing clinical trials, enrollment not feasible due to distance to an open trial)
  • There are no comparable or satisfactory alternative therapy options available

Things to keep in mind:

  • "Investigational new drug" means a new drug or biological drug that is used in a clinical investigation
  • The terms "investigational drug" and "investigational new drug" are deemed to be synonymous
  • Investigational medical products have not yet been approved or cleared by the FDA and the FDA has not found these products to be safe and effective for their specific use
  • The investigational medical product may, or may not, be effective in the treatment of the condition
  • Use of the product may cause unexpected serious side effects
  • The patient and their licensed physician should consider all possible risks
  • The drug Company (Industry) must be willing to provide the investigational medical product and either sponsors the expanded access, allows the FDA to cross-reference to their industry IND (for drugs and biologics) or IDE (medical devices) on behalf of the expanded access sponsor-investigator through the use of a letter of authorization, or provides the necessary investigational medical product information for the sponsor-investigator to submit to support an expanded access request

Licensed physician role:

  • Determine there are no available clinical trials for the patient
  • Confirm patient's current disease or condition qualifies for expanded access
  • Identify the appropriate expanded access request type
  • Confirm industry will provide investigational medical product
  • Facilitate the process
  • Manage the treatment

Categories of expanded access for drugs

For more information, visit the FDA expanded access categories for drugs (including biologics) page.

1. Individual patient

  • Individual patient expanded access IND (also referred to as a single patient IND)
    • See checklist for submitting a request for a single patient IND for a drug or biologic
  • Individual patient expanded access protocol (also referred to as a Single Patient Protocol)
  • Individual patient access in an emergency (also known as emergency expanded access use and emergency access)
    • Please note that emergency INDs and protocols are a subset of individual patient access.

2. Intermediate-size patient population access

  • Intermediate-size patient population expanded access IND
  • Intermediate-size patient population expanded access protocol

3. Expanded access for widespread use

  • Treatment IND
  • Treatment protocol

How to submit a request (non emergency)

Expanded access requests can be submitted on paper or via the FDA expanded access eRequest. The FDA expanded access page provides more information on required forms and step-by-step instructions for different categories of expanded access requests.

*Emergency use

Submit an emergency use request for expanded access to a drug or biologic at Children's Wisconsin.

  • Reference guidance – Individual patient expanded access IND applications (also found in IRBNet) for information on the process/steps for both non-emergency and emergency requests.
  • If there is an emergency that requires a patient to be treated before a written IND submission can be made to FDA, an emergency IND may be granted by FDA. Under the emergency use provisions in the FDA regulations (21 CFR 56.104(c)), the emergency use of an unapproved drug is an exemption from prior review and approval by the IRB, but must be reported to the IRB. The emergency exemption from prospective IRB review allows for one emergency use of a drug or biologic without prospective IRB review. FDA regulations require that any subsequent use of the investigational product at the institution may need prospective IRB review and approval. However, in guidance documents, FDA acknowledges that it would be inappropriate to deny emergency treatment to a second individual if the only obstacle is that the IRB has not had sufficient time to convene a meeting to review the protocol.